Allogeneic stem cell transplant (alloSCT) may be a promising option for patients with treatment-resistant chronic lymphocytic leukemia (CLL), regardless of the patient's underlying genetic abnormalities, according to the results of a study published online in the journal Blood.
While survival rates for leukaemia have generally improved in the last decade, patients with rare, more aggressive forms of CLL do not respond well to standard chemotherapy-based and targeted treatments and often die within a few years of diagnosis.
Patients with CLL who are treatment-resistant have been shown to have genetic abnormalities that predict their lack of response. In this study, researchers investigated whether alloSCT could be an effective treatment for this patient population, independent of underlying genetic abnormalities.
"This study, which is one of the largest of its kind, confirms that allogeneic stem cell transplants are a promising therapeutic option for treatment-resistant CLL patients fighting particularly aggressive disease, regardless of their genetic risk profile," said lead author Peter Dreger, MD, Department of Medicine, University of Heidelberg, Heidelberg, Germany. "However, because stem cell transplants come with serious risks, they should be reserved for only this group of patients until further studies can be done."
The prospective, phase 2 study included a total of 90 patients with treatment-resistant CLL. Patients received alloSCT from either healthy siblings or unrelated, but matched, volunteers.
Prior to the transplant, patients received a reduced-intensity conditioning approach with fludarabine and cyclophosphamide to reduce complications and allow the donor stem cells to fight the disease themselves.
After treatment with alloSCT, more than 40% of the patients enjoyed long-term freedom from relapse.